EU/3/05/280 - orphan designation for treatment of B-cell chronic lymphocytic leukaemia

acadesine

Overview

On 27 May 2005, orphan designation (EU/3/05/280) was granted by the European Commission to Advanced In Vitro Cell Technologies S.L., Spain, for acadesine for the treatment of B-cell chronic lymphocytic leukaemia.

In September 2008, Advanced In Vitro Cell Technologies S.L. changed name to Advancell - Advanced In Vitro Cell Technologies S.A.

For a list of the administrative updates to this public summary of opinion, please refer to the PDF document below.

What is B-cell chronic lymphocytic leukaemia?

B-cell chronic lymphocytic leukaemia is a disease in which cancer cells are found in the blood and the bone marrow. The bone marrow is the spongy tissue inside the large bones in the body. Normally, the bone marrow makes cells called blasts that mature into several different types of blood cell that have specific functions in the body. These include red cells, white cells and platelets. Red blood cells carry oxygen and other materials to all tissues of the body. White blood cells fight infection. Platelets support blood clotting. When leukaemia develops, the bone marrow produces large numbers of abnormal blood cells. There are several types of leukaemia. B-cell chronic lymphocytic leukaemia is a cancer of a type of white blood cells called B-lymphocytes. The lymphocytes multiply too quickly and live too long, so there are too many of them circulating in the blood. These leukaemic lymphocytes look normal, but they are not fully developed and do not work properly. Over a period of time these abnormal cells replace the normal white cells, red cells and platelets in the bone marrow. B-cell chronic lymphocytic leukaemia is the most common type of leukaemia and mainly affects older people and is rare in people under the age of 40. B-cell chronic lymphocytic leukaemia is chronically debilitating and life-threatening due to the severe prognosis and the poor long-term survival for high-risk patients.

What is the estimated number of patients affected by the condition?

At the time of designation, B-cell chronic lymphocytic leukaemia affected approximately 2.7 in 10,000 people in the European Union (EU). This was equivalent to a total of around 126,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

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*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union (EU 25), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 466,600,000 (Eurostat 2005).

What treatments are available?

Treatment for leukaemia is complex and depends on a number of factors including the type of leukaemia, the extent of the disease and whether the leukaemia has been treated before. It also depends on the age, the symptoms, and the general health of the patient. Some people with B-cell chronic lymphocytic leukaemia never have treatment if their illness is not causing any symptoms and is progressing only very slowly. Treatment is only started if and when the symptoms become troublesome. The current main treatment of B-cell chronic lymphocytic leukaemia is chemotherapy (using drugs to kill cancer cells). Several products were authorised or the condition in the Community at the time of submission of the application for orphan-drug designation.

Acadesine could be of potential significant benefit for the treatment of B-cell chronic lymphocytic leukaemia because it might act differently from other medicinal products. This assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

How is this medicine expected to work?

So-called programmed cell death (apoptosis) is a process that is very important for the natural death of cells. In B-cell chronic lymphocytic leukaemia the cells live longer then normal. It is not completely understood how acadesine works in the human body, but it might help to specifically destroy the cancer cells through activation of some specific reactions inside the cancer cell itself, which trigger the apoptosis.

What is the stage of development of this medicine?

The evaluation of the effects of acadesine in experimental models is ongoing.

At the time of submission of the application for orphan designation, no clinical trials in patients with B-cell chronic lymphocytic leukaemia had been initiated.

Acadesine was not marketed anywhere worldwide for B-cell chronic lymphocytic leukaemia or designated as orphan medicinal product elsewhere for this condition, at the time of submission.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 7 April 2005 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria:

• the seriousness of the condition;
• the existence of alternative methods of diagnosis, prevention or treatment;
• either the rarity of the condition (affecting not more than 5 in 10,000 people in the Community) or insufficient returns on investment.

Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

EU/3/05/280: Public summary of positive opinion for orphan designation of acadesine for the treatment B-cell chronic lymphocytic leukaemia

Adopted Reference Number: EMEA/COMP/121542/2005 Rev.2

Key facts

• Active substance:
  acadesine
• Intended use:
  Treatment of B-cell chronic lymphocytic leukaemia
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/05/280
• Date of designation:
  27/05/2005
• Sponsor:

  Advancell - Advanced In Vitro Cell Technologies S.A.; Via Augusta 59, 3rd floor, door 313; 08006 Barcelona; Spain; Tel. [phone]; Fax [phone]